Extract from the Register of European Patents

About this file: EP2799548

EP2799548 - ANTISENSE NUCLEIC ACID [Right-click to bookmark this link]
StatusThe patent has been granted
Status updated on  19.07.2019
Database last updated on 16.10.2019
FormerGrant of patent is intended
Status updated on  14.04.2019
FormerExamination is in progress
Status updated on  12.05.2017
Most recent event   Tooltip19.07.2019(Expected) grantpublished on 21.08.2019  [2019/34]
Applicant(s)For all designated states
Nippon Shinyaku Co., Ltd.
14, Kisshoin Nishinosho Monguchicho Minami-ku Kyoto-shi
Kyoto 601-8550 / JP
For all designated states
National Center of Neurology and Psychiatry
4-1-1, Ogawahigashi-cho
Kodaira-shi
Tokyo 187-8551 / JP
[2019/34]
Former [2014/45]For all designated states
Nippon Shinyaku Co., Ltd.
14, Kisshoin Nishinosho Monguchicho Minami-ku Kyoto-shi
Kyoto 601-8550 / JP
For all designated states
National Center of Neurology and Psychiatry
4-1-1, Ogawahigashi-cho
Kodaira-shi
Tokyo 187-8551 / JP
Inventor(s)01 / WATANABE, Naoki
Room 402 RuvioII
21-3 Sakura 1-chome
Tsukuba-shi Ibaraki 305-0003 / JP
02 / SEO, Haruna
10-14-1401 Senjusekiya-cho
Adachi-ku
Tokyo 120-0024 / JP
03 / TAKEDA, Shin'ichi
c/o National Center of Neurology and Psychiatry
1-1 Ogawahigashi-cho 4-chome
Kodaira-shi Tokyo 187-8551 / JP
04 / NAGATA, Tetsuya
c/o National Center of Neurology and Psychiatry
1-1 Ogawahigashi-cho 4-chome
Kodaira-shi Tokyo 187-8551 / JP
 [2014/45]
Representative(s)HGF Limited
Saviour House
9 St. Saviourgate
York YO1 8NQ / GB
[2019/34]
Former [2014/45]Vossius & Partner
Siebertstrasse 4
81675 München / DE
Application number, filing date12861221.527.12.2012
[2019/34]
WO2012JP84295
Priority number, dateJP2011028804028.12.2011         Original published format: JP 2011288040
JP2012004309229.02.2012         Original published format: JP 2012043092
[2014/45]
Filing languageJA
Procedural languageEN
PublicationType: A1  Application with search report
No.:WO2013100190
Date:04.07.2013
Language:JA
[2013/27]
Type: A1 Application with search report 
No.:EP2799548
Date:05.11.2014
Language:EN
[2014/45]
Type: B1 Patent specification 
No.:EP2799548
Date:21.08.2019
Language:EN
[2019/34]
Search report(s)International search report - published on:JP04.07.2013
(Supplementary) European search report - dispatched on:EP19.10.2015
ClassificationInternational:C12N15/113, A61K31/7088, A61K48/00, A61P21/04, A61P43/00
[2014/45]
Designated contracting statesAL,   AT,   BE,   BG,   CH,   CY,   CZ,   DE,   DK,   EE,   ES,   FI,   FR,   GB,   GR,   HR,   HU,   IE,   IS,   IT,   LI,   LT,   LU,   LV,   MC,   MK,   MT,   NL,   NO,   PL,   PT,   RO,   RS,   SE,   SI,   SK,   SM,   TR [2014/45]
Extension statesBANot yet paid
MENot yet paid
TitleGerman:ANTISENSE-NUCLEINSÄURE[2014/45]
English:ANTISENSE NUCLEIC ACID[2014/45]
French:ACIDE NUCLÉIQUE ANTISENS[2014/45]
Entry into regional phase10.07.2014Translation filed 
10.07.2014National basic fee paid 
10.07.2014Search fee paid 
10.07.2014Designation fee(s) paid 
10.07.2014Examination fee paid 
Examination procedure10.07.2014Examination requested  [2014/45]
10.05.2016Amendment by applicant (claims and/or description)
12.05.2017Despatch of a communication from the examining division (Time limit: M06)
16.11.2017Reply to a communication from the examining division
12.02.2018Despatch of a communication from the examining division (Time limit: M06)
22.08.2018Reply to a communication from the examining division
15.04.2019Communication of intention to grant the patent
09.07.2019Fee for grant paid
09.07.2019Fee for publishing/printing paid
09.07.2019Receipt of the translation of the claim(s)
Divisional application(s)EP19182708.8
The date of the Examining Division's first communication in respect of the earliest application for which a communication has been issued is  12.05.2017
Fees paidRenewal fee
23.12.2014Renewal fee patent year 03
15.12.2015Renewal fee patent year 04
15.12.2016Renewal fee patent year 05
23.11.2017Renewal fee patent year 06
13.12.2018Renewal fee patent year 07
Documents cited:Search[XDI]WO2010048586  (AVI BIOPHARMA INC [US], et al) [XD] 1-4,13,14,16-18,20 * page 7 - page 8 * * page 11 - page 12 * * page 12 - page 14; figures 1, 3, 5, 6 * * page 75 - page 76 * * page 77 - page 78 * * oligonucleotide targeting exons 44 and 45; page 79 - page 82; sequence 34 * * oligonucleotide targeting exon 50; page 89 - page 90; sequences 290, 306, 307 * * oligonucleotides targeting exon 55; page 99 - page 101; sequences 520-569 * * claims 5, 6, 62 * * the whole document * * page 101 - page 103 * [I] 5-12,15,19;
 [XI]WO2010123369  (PROSENSA TECHNOLOGIES BV [NL], et al) [X] 2,4,13-16,20 * oligonucleotides targeting exon 45; page 59 - page 61; figures 1, 2; sequences 76, 115, 127, 135, 557 * * oligonucleotides targeting exon 45; page 72 - page 73 * * oligonucleotides targeting exon 44; page 74 - page 75; sequence 230 * * oligonucleotides targeting exon 50; page 77 - page 78 * * oligonucleotides targeting exon 55; page 80 * * the whole document * [I] 7,8,11,12,17-19;
 [XI]WO2010050801  (PROSENSA TECHNOLOGIES BV [NL], et al) [X] 2,13-16,20 * oligonucleotides targeting exon 45; page 29 - page 30; sequences 3, 49, 57, 65 * * the whole document * [I] 7,8,17-19;
 [XI]US2010168212  (POPPLEWELL LINDA [GB], et al) [X] 2,4,13,14,16-18,20 * page 6 * * oligonucleotides "h45A30/1" and "h45A30/2"; page 10 * * oligonucleotides "h44A30/1" and "h44A30/2" * * the whole document * [I] 7,8,11,12,15,19;
 [X]WO2010050802  (ACADEMISCH ZIEKENHUIS LEIDEN [NL], et al) [X] 2,7,8,13,20 * page 42 - page 43; sequences 479, 487, 495 * * the whole document *;
 [X]WO2009054725  (ACADEMISCH ZIEKENHUIS LEIDEN [NL], et al) [X] 2,4,13,20 * figure 9; sequences 95, 100, 107, 108, 111, 115, 116 * * sequence 266 * * the whole document *;
 [XI]WO2011057350  (UNIV WESTERN AUSTRALIA [AU], et al) [X] 2,13,20 * page 52 - page 53; table 34 * * figure 46A * * the whole document * [I] 7,8,13-19;
 [XI]US7807816  (WILTON STEPHEN DONALD [AU], et al) [X] 3,13-16,20 * column 18; sequence 175 * * the whole document * [I] 9,10,17-19;
 [X]  - STEVE D. WILTON ET AL, "Antisense oligonucleotide-induced exon skipping across the human dystrophin gene transcript", MOLECULAR THERAPY, NATURE PUBLISHING GROUP, GB, vol. 15, no. 7, doi:10.1038/SJ.MT.6300095, ISSN 1525-0024, (20070701), pages 1288 - 1296, (20070206), XP002625829 [X] 2,13,20 * page 1291; table 1 *

DOI:   http://dx.doi.org/10.1038/sj.mt.6300095
 [A]  - A. AARTSMA-RUS ET AL., "Exploring the frontiers of therapeutic exon skipping for Duchenne muscular dystrophy by double targeting within one or multiple exons", MOLECULAR THERAPY, (20060901), vol. 14, no. 3, doi:10.1016/j.ymthe.2006.02.022, ISSN 1525-0016, pages 401 - 407, XP055078028 [A] 1 * the whole document *

DOI:   http://dx.doi.org/10.1016/j.ymthe.2006.02.022
 [A]  - CHRISTOPHE BÉROUD ET AL., "Multiexon skipping leading to an artificial DMD protein lacking amino acids from exons 45 through 55 could rescue up to 63% of patients with Duchenne muscular dystrophy", HUMAN MUTATION, (20070201), vol. 28, no. 2, doi:10.1002/humu.20428, ISSN 1059-7794, pages 196 - 202, XP055046760 [A] 1 * the whole document *

DOI:   http://dx.doi.org/10.1002/humu.20428
 [A]  - MOULTON H.M. AND MOULTON J.D., "Morpholinos and their peptide conjugates: Therapeutic promise and challenge for Duchenne Muscular Dystrophy", BIOCHIMICA ET BIOPHYSICA ACTA (BBA) - BIOMEMBRANES, ELSEVIER, AMSTERDAM, NL, vol. 1798, no. 12, doi:10.1016/J.BBAMEM.2010.02.012, ISSN 0005-2736, (20101201), pages 2296 - 2303, (20100217), XP027430152 [A] 17-19 * the whole document *

DOI:   http://dx.doi.org/10.1016/j.bbamem.2010.02.012
 [A]  - QI-LONG LU ET AL., "The status of exon skipping as a therapeutic approach to Duchenne Muscular Dystrophy", MOLECULAR THERAPY, (20110101), vol. 19, no. 1, doi:10.1038/mt.2010.219, ISSN 1525-0016, pages 9 - 15, XP055076657 [A] 17-19 * the whole document *

DOI:   http://dx.doi.org/10.1038/mt.2010.219
International search[Y]WO2010123369  (PROSENSA TECHNOLOGIES BV [NL], et al);
 [Y]WO2010050802  (ACADEMISCH ZIEKENHUIS LEIDEN [NL], et al);
 [Y]JP2006523101  (ACADEMISCH ZIEKENHUIS LEIDEN);
 [Y]  - AKINORI NAKAMURA ET AL., "Exon skipping therapy for Duchenne muscular dystrophy : outcome of preclinical study and prospective for clinical study", NO TO HATTATSU, (2010), vol. 42, pages 117 - 123, XP008174790
 [Y]  - KOBAYASHI, M. ET AL., "Molecular therapy of muscular dystrophy with exon skipping", JOURNAL OF CLINICAL AND EXPERIMENTAL MEDICINE, (201104), vol. 237, no. 3, pages 251 - 257, XP008174732
 [Y]  - TAKASHI SAITO ET AL., "Development of Exon Skipping Therapy for Duchenne Muscular Dystrophy Using Patient-Derived Cells'', Dai 32 Kai The Japanese Society of Clinical Pharmacology and Therapeutics Nenkai Symposium 2: Hito Soshiki o Mochiita Rinsho Yakurigaku Kenkyu no Hatten", JPN. J. CLIN. PHARMACOL. THER., (20111203), vol. 43, no. 2, pages 91 - 92, XP008174733
 [Y]  - AARTSMA-RUS, A. ET AL., "Antisense-induced exon skipping for duplications in Duchenne muscular dystrophy", BMC MED. GENET., (2007), vol. 8, no. 43, pages 1 - 9, XP002703186

DOI:   http://dx.doi.org/10.1186/1471-2350-8-43
 [Y]  - AARTSMA-RUS, A. ET AL., "Antisense-induced multiexon skipping for Duchenne muscular dystrophy makes more sense", AM. J. HUM. GENET., (2004), vol. 74, pages 83 - 92, XP008084158
 [Y]  - LU, Q. L. ET AL., "The status of exon skipping as a therapeutic approach to duchenne muscular dystrophy", MOL. THER., (2010), vol. 19, no. 1, pages 9 - 15, XP055076657

DOI:   http://dx.doi.org/10.1038/mt.2010.219
 [Y]  - MALUEKA, R. G. ET AL., "Antisense oligonucleotide induced dystrophin exon 45 skipping at a low half-maximal effective concentration in a cell-free splicing system", NUCLEIC ACID THER., (201110), vol. 21, no. 5, pages 347 - 353, XP055076872

DOI:   http://dx.doi.org/10.1089/nat.2011.0310
 [Y]  - HEEMSKERK, H. A. ET AL., "In vivo comparison of 2'-O-methyl phosphorothioate and morpholino antisense oligonucleotides for Duchenne muscular dystrophy exon skipping", J. GENE MED., (2009), vol. 11, pages 257 - 266, XP002541504

DOI:   http://dx.doi.org/10.1002/JGM.1288
 [Y]  - TAKAGI, M. ET AL., "Design of 2'-O-Me RNA/ENATM chimera oligonucleotides to induce exon skipping in dystrophin pre-mRNA", NUCLEIC ACIDS SYMP. SER., (200411), vol. 48, no. 1, pages 297 - 298, XP003031264

DOI:   http://dx.doi.org/10.1093/nass/48.1.297
 [Y]  - AARTSMA-RUS, A. ET AL., "Exploring the frontiers of therapeutic exon skipping for Duchenne muscular dystrophy by double targeting within one or multiple exons", MOL. THER., (2006), vol. 14, no. 3, pages 401 - 407, XP055078028

DOI:   http://dx.doi.org/10.1016/j.ymthe.2006.02.022
 [Y]  - MOULTON, H. M. ET AL., "Morpholinos and their peptide conjugates: therapeutic promise and challenge for Duchenne muscular dystrophy", BIOCHIM. BIOPHYS. ACTA, (2010), vol. 1798, pages 2296 - 2303, XP027430152

DOI:   http://dx.doi.org/10.1016/j.bbamem.2010.02.012
 [Y]  - HU, Y. ET AL., "Guanine analogues enhance antisense oligonucleotide-induced exon skipping in dystrophin gene in vitro and in vivo", MOL. THER., (2010), vol. 18, no. 4, pages 812 - 818, XP009171294

DOI:   http://dx.doi.org/10.1038/mt.2009.320
 [Y]  - PICHAVANT, C. ET AL., "Current status of pharmaceutical and genetic therapeutic approaches to treat DMD", MOL. THER., (201105), vol. 19, no. 5, pages 830 - 840, XP055078033

DOI:   http://dx.doi.org/10.1038/mt.2011.59
 [Y]  - WU, B. ET AL., "Targeted skipping of human dystrophin exons in transgenic mouse model systemically for antisense drug development", PLOS ONE, (201105), vol. 6, no. 5, pages 1 - 11, XP009171295

DOI:   http://dx.doi.org/10.1371/journal.pone.0019906
 [Y]  - AARTSMA-RUS, A. ET AL., "Targeted exon skipping as a potential gene correction therapy for Duchenne muscular dystrophy", NEUROMUSCUL. DISORD., (2002), vol. 12, pages S71 - S77, XP002250906

DOI:   http://dx.doi.org/10.1016/S0960-8966(02)00086-X
 [Y]  - WILTON, S. D. ET AL., "Antisense oligonucleotide-induced exon skipping across the human dystrophin gene transcript", MOL. THER., (200707), vol. 15, no. 7, pages 1288 - 1296, XP002625829

DOI:   http://dx.doi.org/10.1038/sj.mt.6300095
 [A]  - BEROUD, C. ET AL., "Multiexon skipping leading to an artificial DMD protein lacking amino acids from exons 45 through 55 could rescue up to 63% of patients with Duchenne muscular dystrophy", HUM. MUTAT., (2007), vol. 28, no. 2, pages 196 - 202, XP055046760

DOI:   http://dx.doi.org/10.1002/humu.20428
ExaminationEP1568769
by applicantWO2006000057
 WO2004048570
 US2010168212
 WO2010048586
 WO2004083446
 WO2010050801
 WO2009139630
 WO2006129594
 WO2006038608
 WO9109033
 WO2009064471
 JP2924179B
 WO2008096690
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